NCT07097701

An Open-Label, Randomized, Cross-Over Study to Investigate the Efficacy and Safety of Mexiletine PR Compared to Mexiletine IR

An Open-Label, Randomized, Cross-Over Study to Investigate the Efficacy and Safety of Mexiletine PR Compared to Mexiletine IR in Patients With Non-Dystrophic Myotonias (ACHILLES Study)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Lupin Ltd. · Industry
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

Summary

An Open-Label, Randomized, Cross-Over Study to Investigate the Efficacy and Safety of Mexiletine PR compared to Mexiletine IR in Patients with Non-Dystrophic Myotonias (ACHILLES study)

Detailed description

This is a multicenter, open-label, randomized, cross-over study intended to evaluate the efficacy and the safety of mexiletine PR (QD) vs mexiletine IR (TID) in patients with non-dystrophic myotonias including myotonia congenita (MC), paramyotonia congenita (PC) and sodium channel myotonia (SCM). The study will consist of a 4-week screening period followed by two 12-week treatment periods. Eligible patients will be randomized to receive mexiletine PR or mexiletine IR for 12 weeks. After a wash out period of at least 7 days the patients will receive the opposite treatment for 12 weeks. A total of 24 patients are planned to be enrolled (with a target enrollment of 12 naïve to previous mexiletine treatment and 12 previously treated with mexiletine). Safety assessments include patient- and physician-reported adverse event reporting, electrocardiogram (ECG), standard clinical laboratory evaluations, physical examinations, and vital signs. Efficacy assessments include patient-reported outcomes (PROs) and functional capacity outcome measures.

Conditions

• Myotonia

Interventions

Type	Name	Description
DRUG	Granular powder in unit dose foil-lined sachet Mexiletine (PR)	Mexiletine (PR)
DRUG	Oral Capsule Mexiletine (IR)	Mexiletine (IR)

Timeline

• Start date: 2025-09-03
• Primary completion: 2027-01-22
• Completion: 2027-02-22
• First posted: 2025-07-31
• Last updated: 2025-12-22

Locations

1 site across 1 country: Belgium